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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

NCTID NCT04783181 (View at clinicaltrials.gov)
Description
Indication Congenital Adrenal Hyperplasia
Compound Name AAV5 BBP-631 (AAV5-CYP21A2)
Sponsor Adrenas Therapeutics Inc
Funder Type Industry
Status
Active not recruiting
Enrollment Count 8

Therapy Information

Target Gene/Variant CYP21A2
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV5
Editor Type
Dose 1 1.5 x 10^13 vg/kg
Dose 2 3.0 x 10^13 vg/kg
Dose 3 6.0 x 10^13 vg/kg
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2021-03-01
Completion Date 2029-02
Last Update 2024-09-26

Participation Criteria

Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations

No.of Trial Sites 5
Locations United States

Regulatory Information

Has US IND True
Recent Updates Bridge Bio is cutting this program, lack of sufficient efficacy

Resources/Links

News and Press Releases
Preclinical Publications
Protocol